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Safety and Efficacy of Fingolimod in Pediatric Patients With Multiple Sclerosis

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ClinicalTrials.gov Identifier: NCT01892722
Recruitment Status : Recruiting
First Posted : July 4, 2013
Results First Posted : September 19, 2018
Last Update Posted : August 14, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
To evaluate the safety and efficacy of fingolimod vs. interferon beta-1a i.m. in pediatric patients with multiple sclerosis (MS)

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Drug: Interferon beta-1a Drug: Fingolimod Drug: Placebo capsule Drug: Placebo i.m. injection Phase 3

Detailed Description:
The study is divided into a Core Phase, which includes the Double-Blind Treatment Period, and an Extension Phase in which all patients will be treated with fingolimod. The Core Phase is a 24-month, double-blind, randomized, active-controlled, parallel-group multicenter study phase to evaluate the efficacy and safety of fingolimod compared to IFN β-1a in children/adolescent patients aged 10-17 years old with MS. The Extension Phase is a 60-month (5 year) study phase for patients who complete the Core Phase of the study and meet all inclusion/exclusion criteria and for patients who will be recruited in the younger cohort to participate in the Extension Phase. The 'younger cohort' refers to the population of pediatric patients fulfilling any single one or a combination of the following criteria: being ≤12 years of age, or weighing ≤40 kg, or being prepubertal (i.e. pubertal status of Tanner stage <2). The recruitment of the younger cohort (30 patients) was requested as a post- approval health authority commitment

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 245 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: There are 2 arms in the core phase. In the extension phase all participants are receiving open-label.study drug. A third arm has been added to enroll up to 30 new younger patients per HA post approval committment.
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A 2 Year, Double-blind, Randomized, Multicenter, Active-controlled Core Phase to Evaluate Safety & Efficacy of Daily Fingolimod vs Weekly Interferon β-1a im in Pediatric Patients With Multiple Sclerosis and 5 Year Fingolimod Extension Phase
Actual Study Start Date : July 26, 2013
Actual Primary Completion Date : July 14, 2017
Estimated Study Completion Date : December 15, 2028


Arm Intervention/treatment
Experimental: Fingolimod
Fingolimod was administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight) with the aim to achieve systemic exposure in range of that in adults at the licensed 0.5 mg dose. Participants in this arm during core continued into extension and received open-label treatment
Drug: Fingolimod

Administrated orally once daily:

0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.


Drug: Placebo capsule
Matching placebo capsule required for double-dummy masking to blind formulations.

Active Comparator: Interferon beta-1a
An intramuscular (IM) injection of Interferon beta-1a was administered once weekly during core phase. Participants switched to receive open-label fingolimod in extension phase
Drug: Interferon beta-1a
Administration once weekly via i.m. injections.

Drug: Placebo i.m. injection
Matching placebo i.m. injection required for double-dummy masking to blind formulations.

Experimental: Fingolimod-Younger Cohort
The 'younger cohort' refers to the new pediatric patients to be recruited in the extension phase who fulfill any single one or a combination of the following criteria: being ≤12 years of age, or weighing ≤40 kg, or being prepubertal (i.e. pubertal status of Tanner stage <2)
Drug: Fingolimod

Administrated orally once daily:

0.5 mg capsule for patients over 40 kg or 0.25 mg capsule for patients 40 kg or less.





Primary Outcome Measures :
  1. Frequency of Relapses in Patients Treated for up to 24 Months [ Time Frame: 24 months ]
    Frequency of relapses assessed by the annualized relapse rate (ARR). The ARR is defined as the average number of confirmed relapses per year (total number of confirmed relapses divided by the total days in the study multiplied by 365.25).


Secondary Outcome Measures :
  1. New/Newly Enlarged T2 Lesions [ Time Frame: 24 months ]
    Annualized rate of the number of new/newly enlarged T2 lesions up to Month 24

  2. Time to First Relapse [ Time Frame: 24 months ]
    Time to first relapse was determined.

  3. Proportion of Patients Relapse-free [ Time Frame: 24 months ]
    Proportion of patients relapse-free was determined

  4. T1 Gd- Enhancing Lesions [ Time Frame: 24 months ]
    Number of T1 Gd-enhancing lesions per scan up to Month 24

  5. Pharmacokinetics (Cavg) of Fingolimod-P [ Time Frame: 24 months ]
    Cavg (average drug concentration over the dose interval) will be evaluated.

  6. Pharmacokinetic/Pharmacodynamic Relationship for Fingolimod-P to Lymphocyte Levels [ Time Frame: 24 months ]
    Population PK/PD modeling approaches were used to relate the individual fingolimod-P concentrations to lymphocyte counts.



Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria Core Phase:

  • diagnosis of multiple sclerosis
  • at least one MS relapse during the previous year or two MS relapses in the previous 2 years or evidence of Gd enhancing lesions on MRI within 6 months EDSS score of 0 to 5.5, inclusive

Key Exclusion Criteria Core Phase:

  • patients with progressive MS
  • patients with an active, chronic disease of the immune system other than MS
  • patients meeting the definition of ADEM
  • patients with severe cardiac disease or significant findings on the screening ECG.
  • patients with severe renal insufficiency

Key Inclusion Criteria Extension Phase:

Applies to all patients participating in the Core Phase and then entering the Extension Phase. 1. Patients that originally met Core Phase Inclusion criteria and completed the Core phase on or off of study drug.

Applies to patients newly recruited to participate in the Extension Phase.

  • All newly recruited patients' that enroll directly into the Extension Phase must fulfill the local country health authority product label approved for pediatric age group for inclusion criteria.
  • Central review (including initial MRI report) of the diagnosis of pediatric MS will be required for all newly recruited patients.

Key Exclusion Criteria Extension Phase:

Applies to patients who completed the Core Phase, but prematurely discontinued study drug.

  1. Premature discontinuation of the study drug during the Core Phase due to:

    • an adverse event,
    • serious adverse event,
    • laboratory abnormality
    • other conditions leading to permanent study drug discontinuation due to safety reasons
  2. Patients with known new events or concomitant medications (washout periods required prior to Visit 15) that would exclude them from the Core Phase exclusion criteria. Serological or other additional tests will not be required.

Applies to patients newly recruited in the younger cohort to participate in the Extension Phase.

1. All newly recruited patients in the younger cohort that enroll directly into the Extension Phase must fulfill the exclusion criteria for the core phase.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01892722


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 Novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

Locations
Show Show 104 study locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Novartis ( Novartis Pharmaceuticals ):
Statistical Analysis Plan  [PDF] August 4, 2017
Study Protocol  [PDF] November 16, 2016

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01892722    
Other Study ID Numbers: CFTY720D2311
2011-005677-23 ( EudraCT Number )
First Posted: July 4, 2013    Key Record Dates
Results First Posted: September 19, 2018
Last Update Posted: August 14, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
pediatric
multiple sclerosis
fingolimod
pre-pubertal
low weight
children
adolescent
MS
core phase
extension phase
younger cohort
Additional relevant MeSH terms:
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Multiple Sclerosis
Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Interferons
Interferon-beta
Interferon beta-1a
Fingolimod Hydrochloride
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic
Immunosuppressive Agents